|Judy Roberson at podium, Bob Klein, former chairman of the California stem cell agency at left, Claire Pomeroy, former|
UC Davis vice chancellor and agency director at right. UC Davis photo
"Over 40 HD advocates joined the CIRM Stem Cell Champion campaign in April, promoted by Kevin McCormack (the agency's director of communications), in the hopes that pressure from affected families will prompt the FDA to become open to stem cell therapies and allow the increased risks that naturally go along with new therapies.
"For people with Huntington's disease, which is 100 percent fatal and has zero therapies, we are willing to take on more risk since we're dying anyway.
"The FDA has delayed the fully enrolled, CIRM funded, first-in-human clinical trial using adult stem cells at UC Davis with Drs. Vicki Wheelock and Jan Nolta. The NIH RAC committee enthusiastically approved their novel therapeutic clinical trial. Then the FDA asked for additional animal studies, this time with three pigs at a cost of over $330,000 plus two years of additional research; costs will approach $1 million.
"One HD family from New York has funded one of the pigs, but this gap in funding has shelved this promising research!
"Devastated patients and their families see this add-on research requirement as a delay from the FDA, which means that this fully enrolled trial, with 24 patients who meet today's criteria will progress and may not qualify in two to four years even if this project later receives FDA approval to begin a phase one trial. FDA delays are killing us!
"We need a new FDA 2.0 because doing NOTHING is doing harm!" Sphere: Related Content